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FDA approves first gene therapy treatment of Duchenne muscular dystrophy
- Despegar 3y agoIt's unfortunate that biotech funding also depends on the same low rate environment that tech does, because while the tech industry has mostly been spinning its wheels on nonsense after the iPhone (like crypto), there's real innovation in biotech.
- mattigames 3y agoThe us military wastes 2.01 trillion dollars a year, any alignment between ending human suffering and capitalism is merely incidental.
- JumpCrisscross 3y ago> us military wastes 2.01 trillion dollars a year Do you think gene therapies are developed while the scientists are dealing with food insecurity and competing warlords?
- mikepurvis 3y agoIs that what $2T a year is buying? Looking at the wars over the last twenty years, it's far from clear that the presence of the US military has really helped reduce or end conflict.
- JumpCrisscross 3y ago> it's far from clear that the presence of the US military has really helped reduce or end conflict This is difficult to answer. What can be shown is the post-WWII era has been unusually peaceful within the scope of the modern era of nation states. We’re coming up against the century of no great power conflicts of the Metternich Era.
- scarface_74 3y agoThe fact is that US military spending is not to prop up the military. A lot of it is basically just a make work program for civilians. Military leaders have warned for years that one of our biggest threats is our deficit and borrowing money. Every time that the military tries to close unneeded bases and get rid of weapons that aren’t needed, they are thwarted by Congress who doesn’t want to lose jobs in their district. There is a reason that military weapon makers spread out manufacturing throughout the US.
- s1artibartfast 3y agoNot living in a war-torn country like Ukraine where schools and scientists are being hit by missiles? You might make a compelling argument that us defense spending hasn't helped conflict abroad, but the last military conflict of note on us soil was Pearl Harbor in World War II. A reasonable person might conclude that us defense spending has been very effective at preventing military conflicts on us soil. And maybe the case that us defense spending even increases the number of global conflicts if it reduces the chance of conflict at home. The question isn't if the US defense spending increases or decreases total conflict, but for whom it increases or decreases conflict
- johncessna 3y agoThe US is creating that war-torn country so that it can test it's capabilities and weaken Russia at the same time. Your point still stands though, but I don't think in the way you meant it.
- s1artibartfast 3y agoI think it's a valid point and the meaning is clear. If you want me to spell it out, it would be to the advantage of the US military security if every combat capable Ukrainian and Russian were dead. Ukrainian life isn't a threat as much as trivial cost. Given this priority, it should be no surprise that US military policy results in great death and destruction abroad but not at home.
- rfrey 3y agoRussia invaded Ukraine. Unless you think Russia is a US puppet helping the US weaken Russia, I think you should check your prejudices.
- deleted 3y ago[deleted]
- Supermancho 3y ago> Russia invaded Ukraine. Unless you think Russia is a US puppet helping the US weaken Russia, I think you should check your prejudices. The US had been supporting Ukraine before that, both by proxy and explicitly (https://www.state.gov/u-s-security-cooperation-with-ukraine/ https://www.state.gov/u-s-security-cooperation-with-ukraine/). The Russian invasion was incidental to, not a trigger to instigating, the US support of Ukraine. The over-arching behavior, of weakening Russian influence in a region, has been consistent.
- Cthulhu_ 3y agoThe worldwide US military presence has a suppressive effect on wars; I'm confident south-east Asia would have been in multiple wars since WW2 if it wasn't for US presence, and Russia would've tried to take back its former soviet countries if it wasn't for NATO which is mostly the US's $2T army. And I don't think the US has had to deploy its full capacity for a long time, not even Iraq / Afghanistan. That's more of a logistics issue though, they could've multiplied their forces and materials but it wouldn't be any more beneficial.
- BenFranklin100 3y agoThe entire US military budget is approximately $800B. The first clause of your sentence is factually incorrect; the second is a non sequitur.
- johncessna 3y agoNot sure why you're being down voted, here's the data: https://www.usaspending.gov/explorer/budget_function https://www.usaspending.gov/explorer/budget_function Here's what the whitehouse thinks it is, https://www.whitehouse.gov/wp-content/uploads/2023/03/budget_fy2024.pdf https://www.whitehouse.gov/wp-content/uploads/2023/03/budget... (page 63)
- BenFranklin100 3y agoI made a typo which I’ve corrected ($800M —-> $800B).
- twoodfin 3y ago$800B
- BenFranklin100 3y agoCorrect, $800B, not $800M. Thank you for catching the typo. I edited the original post.
- mattigames 3y ago1) That's the amount google returns when asking "What is the US military budged?", forgive me if I wrongly trusted it. 2) Non sequitur? You really believe that spending on war technology (War as in kill-humans-we-dont-like-department) is in no clear way in opposition to spending on health technology? (health as in lets-make-everything-possible-to-make-humans-not-die-department)
- NeuroCoder 3y agoThe first Neuro lab I worked in was funded by the department of defense. Focus was the effects of traumatic brain injury and mechanisms that cause it. It wasnt 2.1 trillion in funding so I can't account for a lot of that I guess
- majormajor 3y agoUS government military spending is all driven by central planning...
- sacnoradhq 3y agoIt's driven by the MIC to buy expensive shit they don't need and doesn't work like the F-35.
- majormajor 3y agoI hardly think lobbying is unique to capitalism. The USSR was famous for political games to win favor with those who allocated things, just like the US military contractor system. Politics are a human issue, not a capitalism one. Saying US military spending reflects poorly on capitalism is just about the dumbest possible anti-capitalist argument. There is so much more low-hanging fruit. Start with non-centrally-managed monopolies and work your way around various nasty corporate practices from there.
- JumpCrisscross 3y ago> unfortunate that biotech funding also depends on the same low rate environment that tech does Maybe this is me peak Chesterton’s fencing it, but there appears to be merit to the peristalsis of exuberant optimism followed by pruning discipline. Absent the first, we wouldn’t have the science. Absent the second, the pressure to push a product diminishes.
- pfisch 3y agoYeah ChatGPT and Stable Diffusion are just a bunch of nonsense...
- mjfl 3y agooverly defensive. kind of proves the point.
- elromulous 3y agoPartially disagree. a lot of bio is jam packed with people pre maturely hyping mouse model success. In that way (the hype machine), it's not that different from tech.
- BenFranklin100 3y agoAs someone who works in the life sciences, I will come to the defense of my colleagues working in tech. A fair amount of life science progress is driven by innovations borne in tech. For instance, deep learning, and specifically GANS, is helping transform image analysis. Also the tech industry is making it possible to process the vast amounts of data produced in the life sciences. Hats off to you guys, great work, seriously. I will say that some aspects of tech (I.e social media driven partisanship, surveillance capitalism, click-back attention stealing advertisement and ‘engagement’) is a (large) net negative, but hopefully over time we will better learn how to manage these negatives.
- Cthulhu_ 3y agoI mean, older tech now, but CAT and CT scans wouldn't have been possible without the huge amounts of processing power and analysis algorithms that the tech industry came up with. One high resolution CT scan generates 350GB of raw data: https://xraylab.esci.umn.edu/frequently-asked-questions#Data https://xraylab.esci.umn.edu/frequently-asked-questions#Data
- jiggunjer 3y agoCAT=CT
- Jorge1o1 3y agoInterestingly, after months of chopping along at the whims of the FDA, the stock actually sold off a little today after the news came out at 2pm.[1] A good example of what it means for news to to be "already priced in," and how in fact, too many traders in a name can overcrowd it and destroy the alpha. It's like game theory. [1] https://yhoo.it/3pbC6aB https://yhoo.it/3pbC6aB
- fullburley 3y agoThe approved label reduced access to 4 and 5 year olds, shrinking the market opportunity in the short term.
- ninja3925 3y agoI somewhat understand your point but the facts imperfectly support it. If information was already priced in, say 90% chance of success, a positive outcome would still provide a lift from the remaining 10%. The decrease sounds like something else is going on.
- mhluongo 3y ago"Buy the rumor, sell the news"
- throwaway2037 3y ago2% is nothing for a single name stock. Who cares. It is just noise. Looking at the close price history here: https://www.google.com/search?q=SRPT+price&rlz=1C1GCEB_enJP1006JP1006&oq=SRPT+price https://www.google.com/search?q=SRPT+price&rlz=1C1GCEB_enJP1... Overall, the last one year has been pretty flat for this stock.
- m3kw9 3y agoIt’s more like ppl sell because they know bull with this environment will keep buying till news hits
- Cthulhu_ 3y agoStock prices have nothing to do with the successes or failures of a company, and everything with hype, marketing, and mainstream appeal. Same with cryptocurrencies and the likes. It's not a rational market, but everyone talks about it like it is.
- hooloovoo_zoo 3y agoRecent Nature article on the treatment https://www.nature.com/articles/d41586-023-01799-z https://www.nature.com/articles/d41586-023-01799-z
- BenFranklin100 3y agoThis is exciting, although the cost of treatment is very high. Contributing factors to the high cost is that gene therapy is a cutting edge approach with a high failure rate and thus high development costs, and DMD is a rare disease with a small patient population. In time, as the development of gene therapy treatments become more refined, costs will go down. Successes in gene therapy treatments are exciting, as the success rate of traditional small molecule therapies have stagnated over the last 10-20 years. Moreover, small molecule therapies may never prove effective in treating many genetic diseases.
- tptacek 3y agoThe disease itself is incredibly expensive. The price of breakthrough treatments will come down faster than the costs of having the disease.
- BenFranklin100 3y agoI don’t know the specifics of the costs of caring for one with DMD, but I strongly agree that in general, new disease therapies are the only sure way to drive down medical costs in the medium and long term. This represents a victory, no doubt.
- fullburley 3y agoIt is very expensive. We will likely have $2M USD in insurance claims for medical this year, and our son is currently aged out of the approved label. At a gross cost of $3.2M for this treatment, its expensive but definitely cost effective relatively speaking.
- arthur2e5 3y agoConsidering this new treatment is just a single dose[^1] that hopefully lasts for quite a while[^2], it might actually cost a lot less in the long run. [^1] see FDA press release and monograph. addditional doses won't do much because by then the immune system has learned to attack the AAV vector -- a different vector strain would be needed and that would be a new drug to be approved [^2] no idea what the turnover rate is. and honestly, the FDA isn't even sure it helps with anything, hence conditional.
- ugh123 3y agoThis treatment targets the dystrophin protein which "helps keep the body’s muscle cells intact". Turns out issues with dystrophin also affect telomere length (at least as studied in the heart of DMD patients [0]). Is this a possible route for natural aging-related research? From [1], "Overall, these data suggest that age-dependent alterations in dystrophin leave the muscle membrane and NMJ more susceptible to contraction-induced damage even before changes in muscle mass are obvious." (tests on rodents) [0] https://med.stanford.edu/cvi/mission/news_center/articles_announcements/2021/single-gene-mutations-and-aging-heart-muscles.html https://med.stanford.edu/cvi/mission/news_center/articles_an... [1] https://academic.oup.com/biomedgerontology/article/72/5/640/2629934 https://academic.oup.com/biomedgerontology/article/72/5/640/...
- Out_of_Characte 3y agoSo far, most age related medicine only come in two forms, those that reduce cell division which improves longevity, and those that increase cell division which improves youthfullness and cancer. Telomeres seem to be a natural counter to reduce aging cells from becoming cancer.
- inciampati 3y agoIt's not so simple. Long and short telomeres can lead to cancer. Short telomeres or their loss can lead to genome rearrangements and other chromosomal problems. This can set the stage for oncogenesis. Long telomeres can allow cells to continue to divide many times. This also can lead to an accumulation of oncogenic drivers. https://www.nejm.org/doi/full/10.1056/NEJMoa2300503 https://www.nejm.org/doi/full/10.1056/NEJMoa2300503 As with many things in biology, maintaining balance is important. Many factors are at play. The message that longer telomeres are better is beguiling, but nature would not make such a simple mistake.
- BillAndrews 3y agoEverytime cells divide they have an increased risk of becoming cancer cells. But not allowing cells to divide is certain death.
- tehjoker 3y ago1. They're using a modified protein 1/4th the size of the regular muscle protein. 2. They only showed they can hit a surrogate endpoint, not a clinical benefit yet. It'll be interesting to see what happens. I hope these kids have better lives as a result of this treatment.
- fullburley 3y agoAs a first of kind treatment, they really didn't pick the right endpoints. Seeing videos of kids able to walk up stairs with confidence, after treatment, when they couldn't before, is rather compelling as evidence. Evidence will also be hard to come by in normal quantities given the disease rarity.
- fullburley 3y agoFor a marketing video that contains both untreated, and before and after DMD with this med, the outcomes are remarkable. https://youtube.com/watch?v=IolcG-a2yeY&feature=share8 https://youtube.com/watch?v=IolcG-a2yeY&feature=share8
- Pxtl 3y agoThis is reassuring. My wife's family has a hyper-rare form of mostly-harmless DMD -- like, every genetic test says my father in law has DMD but he's a healthy octogenarian when most sufferers never saw 30. But it means I always worry about mys son and nephews that may have the gene.
- fullburley 3y agoSerum CK testing would be a reasonable cost available surrogate test, if in normal range than muscle isn't being destroyed as one would see in traditional DMD cases. If high, run the genetic test, free in the US and Canada via Decode Duchenne.
- uptownfunk 3y agoIs this the disease they had the telethon about when we were kids? The Jerry Lewis one, maybe I’m showing my age
- fullburley 3y agoThose were for the Muscular Dystophy Association, who cover DMD along with many other neuromuscular diseases.
- gtsnexp 3y agoGene therapies continue to be delivered using adeno-associated viruses (AAV), which means the essential issue of overexpression hasn't been adequately addressed. This impending clinical trial could very likely result in robust immune responses, potentially necessitating patients to take immunosuppressive drugs for the rest of their lives.
- HoyaSaxa 3y agoI appreciate that most people in this thread are not intimately familiar with this disease, but the casual attitude to it is woefully missing it’s savageness. It unfortunately took my nephew’s good friends life at 13 years of age. You would have never known he had a disease until well past the age of 5. His family provided the most cutting age treatments available including those not yet FDA approved. Yet he deteriorated rapidly between the ages of 10 and 13. For those that don’t know, it is almost impossible to survive past 25 with DMD I cannot speak to the efficacy of this particular drug, but I will optimistically celebrate its approval in hopes that no other family has to watch their child suffer.
- sdflhasjd 3y agoIt's definitely possible to survive past 25, that's just the average life expectancy.
- light_hue_1 3y agoThat doesn't sum it up at all. Without ventilation almost everyone dies by 25 which was the case until the 70s. The poster is right there's a marked decline by your early 20s. And depending on when and where their story happened it could be very accurate: it was almost impossible to make it past 25. Almost everyone needs to be on ventilation to make it further and then the median survival is just shy of 30. That significantly impacts your quality of life unfortunately.
- sdflhasjd 3y agoSorry, I'm unfortunately aware, a friend of mine succumbed to DMD at just 21 even with significant medical care. I was just taking issue with the rather absolute "impossible to survive".
- Traubenfuchs 3y agoI think it would be better for the quality of discourse if people online would stop going into discussions about numbers like this without easily googleable sources. Meta analysis from end of 2021: https://n.neurology.org/content/97/23/e2304 https://n.neurology.org/content/97/23/e2304 "The median survival age from the pre-1970 birth cohort was 18.3 years (95% CI 18.0, 18.9) compared to 24.0 years in the 1970 to 1990 birth cohort (95% CI 22.8, 25.0) and 28.1 years in the post-1990 birth cohort (95% CI 25.1, 30.3)." "The cohort analysis is also likely to represent a comparison of ventilated and nonventilated patients because ventilation was introduced in many clinical settings only in the 1990s."
- aetherspawn 3y agoCould someone clarify, does gene therapy cause a permanent gene mutation that can be passed on to offspring?
- inciampati 3y agoIt could. This is administered via an AAV rh74 vector, an adenovirus isolated from Rhesus monkeys. There is nothing stopping it from getting into the germ line. Notably, this effect may be primarily seen in males, as gametogenesis (cell divisions leading to sperm/eggs) is ongoing throughout life, in contrast to females where gametogenesis has completed at birth.
- deleted 3y ago[deleted]
- stevespang 3y ago[dead]
- adultSwim 3y agoRemember Jesse Gelsinger